New hope for kids with rare immune disease: drug targets root cause
NCT ID NCT05438407
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a drug called leniolisib in children aged 4 to 11 with APDS, a rare genetic condition that causes a faulty immune system. The goal is to see if the drug is safe and can control the disease by blocking an overactive immune signal. About 15 children will take the drug and be monitored for side effects and improvements.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
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Institute of Science Tokyo Hospital
Tokyo, 113-8519, Japan
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Kyoto University Hospital
Kyoto, 606-8507, Japan
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National Institutes of Health
Bethesda, Maryland, 20814, United States
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Necker Hospital Paris
Paris, 75015, France
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Stanford University
Standford, California, 94305-5366, United States
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University of California Los Angeles
Los Angeles, California, 90095, United States
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